On December 09, 2024 Molecular Targeting Technologies, Inc. (MTTI) reported preclinical study results for their proprietary 225Ac-EBTATE against SSTR2 NET cancers online in the European Journal of Nuclear Medicine. ("Long acting 225Ac-EBTATE is highly efficacious against somatostatin receptor-2-positive neuroendocrine tumors" (View Source) (Press release, Molecular Targeting Technologies, DEC 9, 2024, View Source [SID1234648960]).

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Professor Humphrey Fonge of the Université de Laval and lead author commented, "At just 40% of the administered dose reported for 225Ac-DOTATATE, 225Ac-EBTATE produces enhanced anti-tumor efficacy in Small Cell lung cancer (SCLC) and Pan-neuroendocrine tumor (NET) models as shown with complete tumor remissions. At a therapeutic dose of 2x 34 kBq, 225Ac-EBTATE showed general safety for 28 days after blood biochemistry analysis, CBC, and histopathological examination of major organs and tissues. We demonstrated that 225Ac-EBTATE was effective against human small-cell lung cancer (SCLC) with 80% complete remission and 100% survival in preclinical models."

MTTI has licensed commercial use rights for the patented Evans blue (EB) technology from the National Institute of Biomedical Imaging and Bioengineering ("NIBIB"), part of the National Institutes of Health ("NIH"). This platform is the basis for the best-in-class, new generation, collection of targeted radiopharmaceuticals (TRP). EB binds to serum albumin, extending in vivo circulatory half-life and tumor residence time, enhancing up to 30-fold uptake at a tumor and enabling good efficacy with significantly lower radiopharmaceutical activity and fewer dosing cycles vs. the current standard of care. Our 3-year follow up of 177Lu-EBTATE in patients* (N=30) with metastatic neuroendocrine tumors, demonstrated good safety with no nephro- or hepatoxicity and 86% disease control rate using only 40% of the administered dose of 177Lu-DOTATATE.

Dr. Jean-Mathieu Beauregard, MD, Associate Professor of Department of Radiology of Université Laval, said, "I am encouraged to see the positive results of 225Ac-EBTATE in treating small cell lung cancer and Pan-neuroendocrine tumor (NET) in preclinical studies. I look forward to working with MTTI and Professor Fonge to translate this into the clinic."

Dr. Chris Pak, President & CEO of MTTI commented "225Ac-EBTATE effectiveness was encouraging, paralleling the clinical superiority of 177Lu-EBTATE. We look forward to collaborating with Professors Beauregard and Fonge to drive 225Ac-EBTATE into clinical trials in 2025."

On December 9, 2024 TScan Therapeutics, Inc. (Nasdaq: TCRX), a clinical-stage biotechnology company focused on the development of T cell receptor (TCR)-engineered T cell (TCR-T) therapies for the treatment of patients with cancer, reported that updated results from the ongoing ALLOHA Phase 1 trial of TSC-100 and TSC-101 will be presented during an oral session at the 66th American Society of Hematology (ASH) (Free ASH Whitepaper) Annual Meeting and Exposition (Press release, TScan Therapeutics, DEC 9, 2024, View Source [SID1234648995]). TSC-100 and TSC-101 are designed to treat residual disease and prevent relapse in patients with acute myeloid leukemia (AML), acute lymphoblastic leukemia (ALL), and myelodysplastic syndrome (MDS) undergoing allogeneic hematopoietic cell transplantation (HCT) with reduced intensity conditioning.

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"Disease relapse is the leading cause of death in patients undergoing transplant following reduced intensity conditioning and represents a significant unmet medical need," said Chrystal U. Louis, M.D., Chief Medical Officer. "As the majority of patients enrolled in both the treatment and control arms were considered at very high risk for relapse, we are highly encouraged by the preliminary ALLOHA study results, which suggest that TSC-100 and TSC-101 have the potential to eliminate residual disease and prevent relapse in patients with AML, ALL, or MDS post-HCT."

"We are very excited by these data and, based on these results, we intend to launch a pivotal trial in the second half of 2025," said Gavin MacBeath, Ph.D., Chief Executive Officer. "Following recent feedback from the FDA, we believe we have a clear development path and will share our plans at our KOL event tomorrow morning."

In the ongoing ALLOHA Phase 1 trial (NCT05473910), patients receive either TSC-100 or TSC-101 post-HCT, whereas control-arm patients receive HCT alone as per standard of care. To date, 38 patients have been enrolled in the trial and undergone HCT, with 26 in the treatment arm and 12 in the control arm. The key endpoints in the trial are safety and efficacy, with exploratory endpoints including donor chimerism and minimal residual disease (MRD) status.

Key Presentation Highlights:

To date, event-free survival strongly favors the treatment arm (HR=0.30; p=0.04) and early trends suggest a lower probability of relapse (HR=0.28; p=0.14).
2 of 26 (8%) treatment-arm patients relapsed compared to 4 of 12 (33%) control-arm patients. One treatment-arm relapse and subsequent mortality occurred in a very high-risk patient who was taken to transplant without first achieving complete remission, and the other was an extramedullary relapse in the patient’s central nervous system with no evidence of systemic relapse.
Median time to relapse was not evaluable in the treatment arm versus 160 days in the control arm.
8 of 38 (21%) patients in the study had TP53 mutations, with 6 cases in the treatment arm and 2 cases in the control arm. Of the 4 patients in the treatment arm with these mutations who received TCR-T cell infusions, none has relapsed, and one patient has now been relapse-free for 22 months. Of the 2 patients in the control arm with mutated TP53, both relapsed within 6 months of transplant and died shortly thereafter.
TSC-100 and TSC-101 infusions were well-tolerated at all three dose levels with no dose-limiting toxicities. Observed adverse events were similar across the treatment and control arms and were generally consistent with post-HCT adverse events.
TSC-100 and TSC-101 TCR-T cells were detected at all timepoints in all treated patients, including those who have been on study for over a year, with clear evidence of a dose-persistence relationship.
A copy of the presentation materials will be made available on the "Publications" section of the Company’s website at tscan.com once the presentation has concluded.

Virtual Key Opinion Leader (KOL) Event

The Company will host a virtual KOL event featuring Ran Reshef, M.D., M.Sc., on Tuesday, December 10, 2024, at 8:00 a.m. ET to discuss the data presented at ASH (Free ASH Whitepaper), updates with regards to a potential registrational path for the program following its initial meeting with the U.S. Food and Drug Administration, as well as future plans to expand the program, in addition to an update on the Company’s PLEXI-T Phase 1 solid tumor trial.

Dr. Reshef is the Professor of Medicine and Director of the Cellular Immunotherapy Program at Columbia University Irving Medical Center. Details for attending the event can be found here.

On December 9, 2024 Electra Therapeutics, Inc., a clinical stage biotechnology company developing antibody therapies against novel targets for immunological diseases and cancer, reported the presentation of positive results from the completed Phase 1b study of ELA026 for the treatment of secondary hemophagocytic lymphohistiocytosis (sHLH) (Press release, Electra Therapeutics, DEC 9, 2024, View Source [SID1234648897]). ELA026 is a first-in-class monoclonal antibody that targets SIRP-α/β1/γ on the cell surface of myeloid cells and T lymphocytes, the principal pathological immune cells that induce the cytokine storm and hyperinflammation in sHLH. Targeting SIRP to selectively deplete pathogenic immune cells has potential for broad therapeutic applications in immunology, inflammation and cancer. The results of the ELA026 study in sHLH are being presented today at 2:45 PM PST in an oral session at the American Society of Hematology (ASH) (Free ASH Whitepaper) Annual Meeting in San Diego, CA.

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The Phase 1b study was an open-label, single-arm, multicenter study designed to evaluate the safety and efficacy of ELA026, assess the pharmacodynamic (PD) and pharmacokinetic (PK) profile, and identify a dose regimen for further evaluation in a Phase 2/3 study (NCT05416307). The Phase 1b study demonstrated that for sHLH patients with the poorest prognosis, those with malignancy-associated HLH (mHLH), treatment with ELA026 in frontline settings achieved 100% overall response rate by week 4 and 100% hospital discharge, as well as 92% survival at two months. Various PD and HLH-related biomarkers showed that ELA026 rapidly attenuated inflammation, which correlated with clinical responses.

"ELA026 has shown potential to be a transformative treatment for sHLH, a life-threatening disease with no approved therapies. Compared with available treatment, which may include chemotherapy and single cytokine-directed therapies, ELA026 may offer a safer, more effective approach to address the overwhelming immune reaction in sHLH. The Phase 1b study results showing rapid PD and biomarker effects and improved survival at two months are particularly promising," said Swaminathan P. Iyer, MD, Professor in the Department of Lymphoma/Myeloma at The University of Texas MD Anderson Cancer Center. "There is a growing appreciation that controlling the cytokine storm early in the sHLH disease course is necessary to prevent multiorgan failure and allow for proper treatment of the underlying cancer. It is clear that overcoming this critical early period of hyperinflammation in sHLH provide significant clinical benefits for patients."

"We are highly encouraged by these compelling results for sHLH patients, and Electra is working to advance ELA026 into a pivotal study as rapidly as possible. Through rigor and resolve, our team has pioneered a drug development program targeting SIRP, going from a novel idea to pivotal study readiness in five years," said Kathy Dong, PharmD, MBA, President and CEO of Electra Therapeutics. "We have demonstrated the therapeutic value of selectively depleting pathological immune cells to modulate inflammation and look forward to applying this novel approach with ELA026 and our pipeline to expand to other diseases with high unmet need."

Phase 1b Study Results Presented in Oral Session at ASH (Free ASH Whitepaper)
The oral presentation at the ASH (Free ASH Whitepaper) Annual Meeting, entitled "ELA026, a monoclonal antibody targeting signal regulatory protein-α/β1/γ, rapidly controls inflammation and improves 2-month survival in treatment-naïve malignancy-associated hemophagocytic lymphohistiocytosis," is being presented by the lead author, Abhishek Maiti, MD, Assistant Professor in the Department of Leukemia at The University of Texas MD Anderson Cancer Center. This presentation was featured in the section "Granulocytes, Monocytes, and Macrophages: From Inflammation to Hemophagocytic Lymphohistiocytosis." The clinical results presented for ELA026 described the analysis of 12 mHLH patients treated in frontline settings in the completed Phase 1b clinical study. Highlights of the Phase 1b results are as follows:

ELA026 was well tolerated with manageable adverse events.
ELA026 demonstrated rapid PD effects on monocytes and lymphocytes, followed by reduction of markers of inflammation, including ferritin, sCD25, and C‑reactive protein (CRP).
mHLH patients receiving ELA026 in frontline settings achieved treatment outcomes that surpass those observed with available therapies (existing treatments used in the absence of an approved therapy for sHLH):
– 100% overall response rate (12 of 12) ~40% with available therapies.1

– 100% hospital discharge alive (11 of 11, 1 patient withdrew from study before evaluation of this endpoint) inpatient mortality of 20-30% with available therapies.2

– 92% survival at Day 60 (11 of 12) ~50% with available therapies.3

"Because sHLH is a complex, rapidly progressing disease with limited development precedents, advancing ELA026 required a focused approach that our Electra team boldly took on, in collaboration with leading researchers and clinicians. It is gratifying to see the positive Phase 1b results for ELA026 and the potential to improve outcomes for sHLH patients who face this life-threatening condition," said Kim‑Hien Dao, DO, PhD, Chief Medical Officer at Electra. "We have a clear path forward based on recent alignment with the US FDA on the pivotal study design and are moving towards initiating a global Phase 2/3 study in 2025 to address the significant disease burden in sHLH."

About Secondary Hemophagocytic Lymphohistiocytosis (sHLH)
Secondary hemophagocytic lymphohistiocytosis (sHLH) is a rare, life-threatening hyperinflammatory disease for which there is no approved treatment. It can be triggered by cancer, infection, autoimmune disease, or immunotherapy. sHLH is associated with a severe inflammatory response for which patients require immediate intervention. Without treatment, patients may experience multiorgan failure and death. sHLH is associated with high mortality early in the disease course, with malignancy-associated HLH (mHLH) patients having a mortality rate of approximately 50% at two months with available therapies.

On December 9, 2024 SELLAS Life Sciences Group, Inc. (NASDAQ: SLS) ("SELLAS" or the "Company"), a late-stage clinical biopharmaceutical company focused on the development of novel therapies for a broad range of cancer indications, reported additional data from the expansion cohorts in the Phase 2 trial of SLS009, a highly selective CDK9 inhibitor, in relapsed/refractory acute myeloid leukemia (r/r AML) (Press release, Sellas Life Sciences, DEC 9, 2024, View Source [SID1234648913]).

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"We are highly encouraged by the emerging data, which continue to show the potential of SLS009 to transform outcomes of these heavily pretreated AML patients," said Angelos Stergiou, MD, ScD h.c., President and Chief Executive Officer of SELLAS. "In the Cohort 3, the optimal dosing regimen of 30 mg BIW, in patients relapsed or refractory to venetoclax-based regimens, the median overall survival has not been reached but exceeds 7.7 months at latest follow-up, marking a significant milestone for patients in this setting, where the expected mOS is historically around 2.5 months. In addition, we are seeing more than 50% ORR to date in our expansion cohorts in patients with AML-myelodysplasia-related changes (AML-MRC) with ASXL1 mutation and mutations and cytogenic changes other than ASXL1, similar to the previously reported ORR in Cohort 3. We set up an aggressive threshold of 33% response rate before the trial started, and to date we achieved 56% in 5/9 evaluable patients. The rapid enrollment in the expansion cohorts further highlight the critical need for new treatments for our target patient population. These results support the potential of SLS009 to become an important new therapeutic option for this underserved patient population."

Key Highlights from the updated topline data:

As of December 4, 2024, data cutoff, 14 patients were enrolled in Cohort 3 and 14 in Cohort 4 and 5, of which 9 were evaluable at the time of analysis.
At latest follow-up, the mOS has not been reached yet but has exceeded 7.7 months in Cohort 3. This is particularly significant as the expected mOS for patients in this setting is typically 2.5 months.
In expansion cohorts 4 and 5, in patients with AML-myelodysplasia-related changes (AML-MRC) with ASXL1 mutation (cohort 4) and mutations and cytogenic changes other than ASXL1 (cohort 5) the ORR was 56% in 9 evaluable for efficacy patients.
SLS009 was well-tolerated with no new safety signals observed to date as the regimen remains safe in additional patients enrolled to date.
The Phase 2 clinical trial of SLS009 is an open-label, single-arm, multi-center study designed to evaluate the safety, tolerability, and efficacy of SLS009 in combination with venetoclax and azacitidine at two dose levels, 45 and 60 mg. In the 60 mg dose cohort patients were randomized into either a 60 mg dose once per week or a 30 mg dose two times per week. The trial was expanded to include two additional cohorts, one with ASXL1 mutated AML patients and one with patients with myelodysplasia-related molecular abnormalities other than ASXL1. In addition to response and survival analyses, the study aims to identify biomarkers for the target patient population and enrichment for further trials. For more information on the study, visit clinicaltrial.gov identifier NCT04588922.

On December 9, 2024 Poseida Therapeutics, Inc. (Nasdaq: PSTX), a clinical-stage allogeneic cell therapy and genetic medicines company advancing differentiated non-viral treatments for patients with cancer, autoimmune and rare diseases, reported interim clinical data from its Phase 1 trial of P-BCMA-ALLO1 in patients with relapsed/refractory multiple myeloma (RRMM), including new profiling of patient responses from Arm C, an optimized lymphodepletion arm (Press release, Poseida Therapeutics, DEC 9, 2024, View Source [SID1234648929]). The P-BCMA-ALLO1 data are being presented, along with two additional Company poster presentations covering new preclinical data for P-CD19CD20-ALLO1 and a patient case study demonstrating the reactivation of a Poseida autologous CAR-T therapy with a T-cell engager, at the 66th ASH (Free ASH Whitepaper) Annual Meeting and Exposition being held in San Diego on December 7-10, 2024.

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P-BCMA-ALLO1 is an investigational non-viral, stem cell memory T cell (TSCM)-rich allogeneic CAR-T cell therapy in Phase 1/1b clinical development for the treatment of patients with RRMM. P-CD19CD20-ALLO1 is an investigational, non-viral TSCM-rich allogeneic CAR-T cell therapy in Phase 1 clinical development for the treatment of patients with B-cell malignancies and is the Company’s first dual CAR-T program. P-BCMA-ALLO1 and P-CD19CD20-ALLO1 are being developed in collaboration with Roche.

"We continue to gain confidence in the potential for P-BCMA-ALLO1 in multiple myeloma, including from the additional sub-analysis of the Phase 1 data presented at ASH (Free ASH Whitepaper)," said Kristin Yarema, Ph.D., President and Chief Executive Officer of Poseida Therapeutics. "We believe the data to-date provide strong validation for our allogeneic cell therapy platform, laying the groundwork for us to extend our non-viral, TSCM-rich approach and drive value with additional clinical programs. This includes P-CD19CD20-ALLO1, our first dual CAR-T supported by preclinical data presented at ASH (Free ASH Whitepaper), and with clinical data anticipated in 2025."

P-BCMA-ALLO1 Phase 1 Data
The poster presentation will highlight Phase 1 clinical data first presented at the 21st International Myeloma Society (IMS) Annual Meeting in September 2024. The data showed a 91% overall response rate (ORR) in Arm C (an optimized lymphodepletion arm), including a 100% ORR in B-cell maturation antigen (BCMA)-naïve patients, and an 86% ORR in those who had received at least one prior BCMA- and/or G protein-coupled receptor class C group 5 member D (GPRC5D)-targeting treatment modality, along with differentiated safety results with no dose-limiting toxicities, low rates of cytokine release syndrome (CRS) and immune effector cell neurotoxicity syndrome (ICANS), all Grade 2 or less, and no graft vs. host disease or Parkinsonism. No patients required anti-myeloma bridging therapy or prophylaxis with steroids or tocilizumab, and there was no invasive apheresis; an average manufacturing wait time, from treatment decision to clinical response, was only 3.5 weeks1 (with a median time to response of 16 days post initial P-BCMA-ALLO1 therapy). The patients in this study had more advanced disease than the myeloma patients studied in clinical trials of approved autologous CAR-T therapies2, and in the intent-to-treat population, 100% of patients were infused with P-BCMA-ALLO1.

New profiling of patient responses from Arm C are included in the ASH (Free ASH Whitepaper) poster presentation. The data from this analysis show consistent P-BCMA-ALLO1 cellular expansion and persistence across different subgroups, including patients that are typically more challenging to treat. Key highlights suggest that P-BCMA-ALLO1:

Cellular kinetics were not impacted by prior BCMA/GPRC5D-targeted therapy
Expands and persists in patients with extramedullary disease (EMD)
P-CD19CD20-ALLO1 Preclinical Data
Preclinical data has demonstrated that P-CD19CD20-ALLO1 delivers high in vitro potency and strong in vivo antitumor activity for either CD19 or CD20 single-positive target cells, as well as double-positive targets. New preclinical data included in the poster presentation show that compared to CD19-single targeting or CD20-single targeting CAR-T cells, P-CD19CD20-ALLO1:

Achieved higher and more durable killing of tumor cells over three rechallenges, even in the presence of only one tumor antigen
Exhibited higher cytotoxicity
Produced higher and more sustained levels of effector cytokines (IL-2, IFN-γ, sFasL, Granzyme A and Granulysin) that play an important role mediating the immune system response to cancers
Showed higher in vivo antitumor efficacy than the CD19-single targeting CAR-T cells
The Company’s P-CD19CD20-ALLO1 Phase 1 clinical trial is enrolling patients with selected B-cell malignancies, with initial clinical data anticipated in 2025.

CAR-T Reactivation with T-cell Engager Case Study
The case study highlights the reactivation of an autologous Poseida CAR-T therapy with a T-cell engager in a patient with relapsed multiple myeloma. The patient attained and remained in stringent complete response over 12 months after CAR-T reactivation. This case highlights the potential of Poseida’s TSCM-based CAR-T therapies to deliver a strong anti-myeloma response with long-term remission and CAR-T cell persistence. The Company believes this is the first time that a T-cell engager has been seen to reactivate a CAR-T therapy.

ASH 2024 Poster Presentations

Title: Late Polyclonal P-BCMA-101 CAR-T Cell Re-expansion and Rapid Complete Response in a Patient with Relapsed Multiple Myeloma Treated with One Cycle of Talquetamab, More Than 3 Years After CAR-T Infusion

Presenting Author: Anupama Kumar, M.D., Assistant Professor, Hematology, Blood & Marrow Transplant, and Cellular Therapy (HBC) Program, University of California, San Francisco (UCSF)
Session: 704. Cellular Immunotherapies: Early Phase Clinical Trials and Toxicities: Poster I
Presentation Date/Time: Saturday, December 7, 2024, 5:30-7:30 p.m. PT (8:30-10:30 p.m. ET)
Room: Halls G-H, San Diego Convention Center
Abstract Number: 2083
Title: P-CD19CD20-ALLO1: Potent Fully Allogeneic CAR-T Therapy Targeting CD19 and CD20 with Superior Efficacy Over Single-Target Products

Presenting Author: Samy Jambon, Ph.D., Poseida Therapeutics
Session: 702. CAR-T Cell Therapies: Basic and Translational: Poster III
Presentation Date/Time: Monday, December 9, 2024, 6:00-8:00 p.m. PT (9:00-11:00 p.m. ET)
Room: Halls G-H, San Diego Convention Center
Abstract Number: 4805
Title: A Phase 1 Study of P-BCMA-ALLO1, a Non-viral, Allogeneic BCMA Directed CAR-T in Relapsed/Refractory Multiple Myeloma (RRMM): Results from Optimized Lymphodepletion Cohort

Presenting Author: Caitlin Costello, M.D., Professor of Medicine, Director of Multiple Myeloma Program, Division of Blood and Marrow Transplant, Moores Cancer Center, University of California, San Diego (UCSD)
Session: 704. Cellular Immunotherapies: Early Phase Clinical Trials and Toxicities: Poster III
Presentation Date/Time: Monday, December 9, 2024, 6:00-8:00 p.m. PT (9:00-11:00 p.m. ET)
Room: Halls G-H, San Diego Convention Center
Abstract Number: 4828
About P-BCMA-ALLO1
P-BCMA-ALLO1 is an allogeneic CAR-T product candidate licensed to Roche targeting B-cell maturation antigen (BCMA) for the treatment of relapsed/refractory multiple myeloma. This allogeneic program includes a VH-based binder that targets BCMA, and interim clinical data presented at IMS in September 2024 support the Company’s belief that T stem cell (TSCM)-rich allogeneic CAR-Ts have the potential to offer effective, safe and reliable treatment addressing unmet needs in multiple myeloma. The FDA has granted P-BCMA-ALLO1 Orphan Drug designation for multiple myeloma and Regenerative Medicine Advanced Therapy (RMAT) designation for adult patients with relapsed/refractory multiple myeloma after three or more prior lines of therapies including a proteasome inhibitor, an immunomodulatory agent and an anti-CD38 antibody.

P-BCMA-ALLO1 is currently being evaluated in a Phase 1/1b trial in patients with multiple myeloma. Additional information about the trial is available at www.clinicaltrials.gov using identifier: NCT04960579.

About P-CD19CD20-ALLO1
P-CD19CD20-ALLO1 is an allogeneic CAR-T cell therapy product candidate being developed for relapsed or refractory B-cell malignancies in partnership with Roche. P-CD19CD20-ALLO1 expresses two fully functional CAR molecules to target cells that express either CD19 or CD20. The dual targeting approach employed in P-CD19CD20-ALLO1 aims to overcome the antigen escape limitations of CD19-only targeted CAR-T therapies by simultaneously targeting both CD19 and CD20. In addition to the dual targeting, P-CD19CD20-ALLO1 uses a novel CD19 binder that showed greater potency in in vivo preclinical models when compared to the canonical FMC63 Single-chain variable fragment (scFv) binder. P-CD19CD20-ALLO1 is an off-the-shelf CAR-T therapy for which patients do not have to undergo apheresis and wait for cells to be manufactured, which can potentially overcome the limitation of autologous CAR-T therapies associated with significant manufacturing times. P-CD19CD20-ALLO1 is being studied in a Phase 1 study in B-cell malignancies (www.clinicaltrials.gov using identifier: NCT06014762). Building on the transformative potential of the CAR-T modality beyond oncology, the Company has recently submitted investigational new drug (IND) applications to the U.S. Food and Drug Administration (FDA) to investigate this program’s potential for patients with multiple sclerosis and systemic lupus erythematosus.